FDA grants clearance to IND for MCO-010 gene therapy in Stargardt patients

Nanoscope Therapeutics Inc. announced today that it has received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.

“This is another important milestone for Nanoscope, giving us the opportunity of conducting a second clinical trial in the U.S. of our proprietary MCO gene therapy platform, designed to restore vision for patients suffering from retinal degenerative diseases,” Sulagna Battacharya, CEO of Nanoscope, said in a statement. “Currently there are no approved treatments for these conditions.”

According to the company, the Phase 2 clinical trial for Stargardt is expected to begin in H1-2022. Nanoscope is currently conducting a Phase 2b multicenter, randomly assigned sham-controlled and double-masked study of MCO-010 for RP patients. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt.

Stargardt is a macular degeneration (MD) affecting children and adults. The company noted that progress on Nanoscope’s Stargardt program trial has implications for MCO as a platform and its impact beyond rare diseases.

David S. Boyer, MD, Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with Keck School of Medicine at the University of Southern California, noted that current

existing trials attempt to slow down the progression of vision loss in patients with Stargardt disease, Optogenetic approach is to restore vision.

“This can be a groundbreaking attempt to evaluate optogenetic gene therapy to improve vision in Stargardt patients,” Boyer said. “I’m excited by the potential MCO-010 has to restore vision for many patients with sight loss caused by outer retinal dystrophies including dry age-related macular degeneration.”

Stargardt is an inherited retinal disease involving dysfunction/degeneration of photoreceptors. Nanoscope’s MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive. It uses proprietary AAV2 vectors to deliver the MCO genes into the cells where they express polychromatic opsins enabling vision in different color environments. The therapy, involving a single intravitreal injection given in a medical office setting, is applicable for RP and Stargardt regardless of underlying gene mutations.